Pooling Knowledge for Neglected Diseases

Open Innovation Provides Access to IP to Save Lives in the Developing World

The Pool for Open Innovation against Neglected Tropical Diseases gives companies access to intellectual property from various pharmaceutical firms, biotech companies, and academic institutions. The goal is to allow important connections in knowledge to be made and, ultimately, to improve healthcare for those living in the developing world.

Ithemba means “hope” in the South African Zulu language, and that is just what iThemba Pharmaceuticals wants to create. The company’s mission is to conduct world-class drug discovery and development of new treatments for neglected infectious diseases, including one that is particularly virulent in South Africa—tuberculosis.

Current therapies for many diseases of the developing world are inaccessible, outdated, unsafe, ineffective, or simply have not yet been created. The lack of a commercial market for these products—as well as challenging development issues—make it difficult for companies to commit typically scarce resources to research and development for neglected tropical diseases.

That’s where the Pool for Open Innovation against Neglected Tropical Diseases comes in. The Pool for Open Innovation, administered by BIO Ventures for Global Health, facilitates access to the compounds, technologies, and expertise that will help organizations like iThemba conduct research on treatments for neglected diseases more efficiently and effectively.

With start-up funds from the South African Technology Innovation Agency, iThemba has developed a strong foundation in synthetic, natural product, and medicinal chemistry. Armed with this burgeoning expertise as well as a desire to move more projects forward in infectious diseases that affect the South African population, iThemba was the first company to sign on as a user of the Pool for Open Innovation.

The Pool aims to motivate innovative and efficient drug discovery and development by opening access to intellectual property or know-how in neglected tropical disease research. It creates the unique opportunity for companies to leverage assets generated by multinational pharmaceutical companies like GlaxoSmithKline (GSK) and proprietary platform-based biotech companies such as Alnylam Pharmaceuticals, as well as academic institutions like the Massachusetts Institute of Technology.

The Pool for Open Innovation’s approach to intellectual property was particularly attractive to iThemba because it goes beyond the data that is already available in the public domain as published patents.

“For instance, the data in the GSK patent list covers both pending and granted applications. The combined information, which was generated through R&D expenditure stretching to many millions of pounds, has tremendous value,” says David Walwyn, iThemba’s CCO. “But the value is unlikely to be in the direct application of an isolated patent; it is more likely to be realized through linking the GSK patents to knowledge in other areas. Access to the full list through the Pool enables these important connections to be made.”

iThemba is partnering with the Pool’s first academic user, the Emory Institute of Drug Discovery (EIDD), led by Dennis Liotta, Ph.D., director of the EIDD and distinguished Emory professor of organic chemistry. To begin this collaboration, scientists at iThemba and Emory took the time to explore the small molecules that GSK contributed to the Pool. This initial screening allowed the companies to identify relevant molecules that suited their expertise and, ultimately, allowed them to pursue two related targets for tuberculosis—malate synthase and isocitratelyase.

When the project began, Dr. Liotta and his colleagues were eager to sift through the hundreds of issued patents and patent applications in the Pool for Open Innovation, identifying those that fit with their expertise in polymerase inhibitors; they then met with GSK to glean insights not captured in the patent text.

“We ultimately got it down to about six groups of patent families that seemed attractive,” says Dr. Liotta.“We wrote up our own graphical summary of what the patents covered and sent it to GSK for review. Then, when we met with GSK in Cape Town, they gave us an update on all those areas in terms of where they were, what they were doing, whether they were actively pursuing any projects, or whether they had stopped.”

For iThemba, the Pool for Open Innovation against Neglected Tropical Diseases offered the opportunity to work with some of the foremost organizations in the world at a critical juncture in its discovery work.

“This partnership is very important to us, especially the opportunity for our scientists to collaborate with some of the top minds in the field and to learn how they do things and how that might affect our own work” says Walwyn.

For Emory, it offered a way to enter a new space in global health research and development, and the opportunity to collaborate with industry where cutting-edge facilities—such as those at GSK’s Tres Cantos Medicines Development Campus in Spain—are far beyond the resources available in academia.

The Pool for Open Innovation also supports new drug development by providing access to novel technologies that address the specific challenges of new target identification and validation for diseases of the developing world. Rachel Meyers, Ph.D., vp of research and RLD at Alnylam, explains how its RNA interference technology contribution could be used for neglected tropical diseases.

“With RNAi, we know we can very quickly get through target validation both in vitro and in vivo, and that’s likely to be a big, quick win for these programs,” she says. “This requires both intellectual property and know-how that Alnylam scientists will provide to relevant parties. We want to enable companies to do some of the work and understand the power of it themselves.”

Ultimately, the Pool is beneficial for contributors and users, as well as a tool for partnering. Nick Cammack, vp and head of GSK’s Tres Cantos Medicines Development Campus, knows that many researchers may have stopped an exciting piece of work because they either couldn’t think how to evolve it further or the opportunity to develop it further didn’t exist.

“Many others in the external world have great ideas and they want to work alongside GSK to pursue the idea that they’ve had while looking at the patent,” says Cammack. “The scientists at GSK would be happy to do that, because it’s their hard work; and, it would help kick-start the ideas for those that have looked at the patents.”

“We believe that facilitating access to intellectual property and patent know-how in a flexible way that appeals to academic and private sector users as well as industry contributors will encourage an entirely new model for collaboration in global health R&D,” says Melinda Moree, CEO of BIO Ventures for Global Health.

“The partnerships created through the Pool for Open Innovation against Neglected Tropical Diseases will move medicines for neglected diseases forward more quickly and effectively and help us reach the Pool’s ultimate goal, to save lives in the developing world.”

Rianna Stefanakis (rsefanakis@bvgh.org) is manager of research and policy, and Don Joseph is COO, both at BIO Ventures for Global Health. To take advantage of the resources or contribute IP to the Pool for Open Innovation against Neglected Tropical Diseases, please visit the Pool website at www.ntdpool.org.

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